Skip to ContentSingle Escalating Dose Pilot Trial of Canakinumab (ILARIS®) in Duchenne Muscular Dystrophy - Parent Project Muscular Dystrophy
Single Escalating Dose Pilot Trial of Canakinumab (ILARIS®) in Duchenne Muscular Dystrophy
The study is an open-label, single ascending dose study to assess safety and to evaluate short-term changes in biomarkers. The first 3 boys will receive canakinumab at a dose of 2 mg/kg, and the second 3 will receive a dose of 4 mg/kg. The study is comprised of four visits: a screening visit with baseline lab assessment, treatment day, a 10-14 day post-treatment evaluation with safety labs, and a 30 day post-treatment evaluation with safety labs. There will be a phone assessment on day 3-5 after treatment to screen for any side effects.
Clinical evaluation will screen for clinical AEs and SAEs. If dose escalation (4 mg/kg) must be terminated due to dose-limiting toxicities, the remaining subjects may be enrolled to evaluate the safety of canakinumab at the lower dose level (2 mg/kg).
One blood draw will occur after consent at least 48 hours prior to treatment day and on days 10-14 and 30 after the canakinumab injection to screen for laboratory abnormalities and collect serum for biomarker analysis. After obtaining consent, prior to treatment, 2 red top and 1 purple top 4 cc tubes will be obtained for screening labs and serum biomarker collection. Tuberculosis screening (Quantiferon-Gold) will use 4 blood tubes (grey, yellow, purple, green) with 1 cc of blood in each. At the following 2 visits with blood draws, two red top and one purple top collection tubes of 4 cc will be collected per subject. For red top blood collection tubes, one tube will be sent to the clinical lab for safety lab processing. The second tube will be processed in the research lab for serum biomarkers.
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A Gene Transfer Therapy to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Therapeutic Plasma Exchange (Plasmapheresis) in Participants With Duchenne Muscular Dystrophy (DMD) and Pre-existing Antibodies to AAVrh74
Actively Recruiting
4 Years to <9 Years
Restoring or Replacing Dystrophin
Actively Recruiting
Therapeutic Approach:Restoring or Replacing Dystrophin
Variant Requirement:Duchenne - Excluding any deletion of exon 8 and/or 9
A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)
Actively Recruiting
2 Years to <5 Years
Restoring or Replacing Dystrophin
Actively Recruiting
Therapeutic Approach:Restoring or Replacing Dystrophin
Variant Requirement:Duchenne - variants in exons 18 to 58
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)
Active, Not Recruiting
8 Years to 18+ Years
Restoring or Replacing Dystrophin
Active, Not Recruiting
Therapeutic Approach:Restoring or Replacing Dystrophin
Variant Requirement:Duchenne - Excluding any deletion of exon 8 and/or 9
A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
Active, Not Recruiting
2+ Years
Restoring or Replacing Dystrophin
Active, Not Recruiting
Therapeutic Approach:Restoring or Replacing Dystrophin
Variant Requirement:Duchenne - variant criteria varies by cohort
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
Active, Not Recruiting
4 Years to <17 Years
Restoring or Replacing Dystrophin
Active, Not Recruiting
Therapeutic Approach:Restoring or Replacing Dystrophin
Variant Requirement:Duchenne - amenable to exon 51 skipping